Low-Risk Neuroblastoma
Intermediate-Risk Neuroblastoma
High-Risk Neuroblastoma
Progressive/Recurrent Neuroblastoma

A link to a list of current clinical trials is included for each treatment section. For some types or stages of cancer, there may not be any trials listed. Check with your doctor for clinical trials that are not listed here but may be right for you.

Treatment of low-risk neuroblastoma may include the following:

• Surgery followed by watchful waiting.
• Watchful waiting alone for certain infants.
• Surgery followed by chemotherapy, when less than half of the tumor is removed or when serious symptoms cannot be relieved by surgery.
• Radiation therapy to treat tumors that are causing serious problems and do not respond quickly to chemotherapy.
• Low- dose chemotherapy.

Treatment of intermediate-risk neuroblastoma may include the following:

• Chemotherapy.
• Chemotherapy followed by surgery and/or radiation therapy.
• Radiation therapy to treat tumors that are causing serious problems and do not respond quickly to chemotherapy.

Treatment of high-risk neuroblastoma may include the following:

• High-dose chemotherapy followed by surgery to remove as much of the tumor as possible.
• Radiation therapy to the tumor site and, if needed, to other parts of the body with cancer.
• Stem cell transplant.
• Chemotherapy followed by 13-cis retinoic acid.
• A clinical trial of monoclonal antibody therapy after chemotherapy.
• A clinical trial of radiation therapy with radioactive iodine before stem cell transplant.
• A clinical trial of stem cell transplant followed by 13-cis retinoic acid.

Patients First Treated for Low-Risk Neuroblastoma

Treatment for recurrent neuroblastoma that is found in one place in the body may include the following:

• Surgery followed by watchful waiting or chemotherapy.
• Chemotherapy.
• High-dose chemotherapy, stem cell transplant, and 13-cis retinoic acid.

Treatment for recurrent neuroblastoma that has spread to other parts of the body may include the following:

• Watchful waiting.
• Surgery followed by chemotherapy.
• Chemotherapy.
• High-dose chemotherapy, stem cell transplant, and 13-cis retinoic acid.
• A clinical trial of a new treatment.

Patients First Treated for Intermediate-Risk Neuroblastoma

For recurrent neuroblastoma that is found in one place in the body, treatment is usually surgery, with or without chemotherapy.

For recurrent neuroblastoma that has spread to other parts of the body, treatment is usually high-dose chemotherapy, stem cell transplant, and 13-cis retinoic acid.

Patients First Treated for High-Risk Neuroblastoma

Treatment of recurrent neuroblastoma in patients first treated for high-risk neuroblastoma may include the following:

• A clinical trial of chemotherapy followed by monoclonal antibody therapy.
• A clinical trial of radiation therapy with radioactive iodine, alone or before stem cell transplant.
• A clinical trial of stem cell transplant.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with neuroblastoma ¹. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ².

Glossary Terms

A drug that is used in the treatment of acne and psoriasis and is being studied in cancer prevention. It is a type of retinoid. Also called isotretinoin.

A term for diseases in which abnormal cells divide without control and can invade nearby tissues. Cancer cells can also spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is cancer that starts in blood-forming tissue such as the bone marrow, and causes large numbers of abnormal blood cells to be produced and enter the blood. Lymphoma and multiple myeloma are cancers that begin in the cells of the immune system. Central nervous system cancers are cancers that begin in the tissues of the brain and spinal cord.

Treatment with drugs that kill cancer cells.

A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical study.

The amount of medicine taken, or radiation given, at one time.

An intensive drug treatment to kill cancer cells, but that also destroys the bone marrow and can cause other severe side effects. High-dose chemotherapy is usually followed by bone marrow or stem cell transplantation to rebuild the bone marrow.

A type of protein made in the laboratory that can locate and bind to substances in the body, including tumor cells. There are many kinds of monoclonal antibodies. Each monoclonal antibody is made to find one substance. Monoclonal antibodies are being used to treat some types of cancer and are being studied in the treatment of other types. They can be used alone or to carry drugs, toxins, or radioactive materials directly to a tumor.

Cancer that arises in immature nerve cells and affects mostly infants and children.

The use of high-energy radiation from x-rays, gamma rays, neutrons, protons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy), or it may come from radioactive material placed in the body near cancer cells (internal radiation therapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Also called irradiation and radiotherapy.

A radioactive form of iodine, often used for imaging tests or to treat an overactive thyroid, thyroid cancer, and certain other cancers. For imaging tests, the patient takes a small dose of radioactive iodine that collects in thyroid cells and certain kinds of tumors and can be detected by a scanner. To treat thyroid cancer, the patient takes a large dose of radioactive iodine, which kills thyroid cells. Radioactive iodine is also used in internal radiation therapy for prostate cancer, intraocular (eye) melanoma, and carcinoid tumors. Radioactive iodine is given by mouth as a liquid or in capsules, by infusion, or sealed in seeds, which are placed in or near the tumor to kill cancer cells.

Cancer that has recurred (come back), usually after a period of time during which the cancer could not be detected. The cancer may come back to the same place as the original (primary) tumor or to another place in the body. Also called recurrent cancer.

Cancer that has recurred (come back), usually after a period of time during which the cancer could not be detected. The cancer may come back to the same place as the original (primary) tumor or to another place in the body. Also called recurrence.

The extent of a cancer in the body. Staging is usually based on the size of the tumor, whether lymph nodes contain cancer, and whether the cancer has spread from the original site to other parts of the body.

A method of replacing immature blood-forming cells that were destroyed by cancer treatment. The stem cells are given to the person after treatment to help the bone marrow recover and continue producing healthy blood cells.

A procedure to remove or repair a part of the body or to find out whether disease is present. An operation.

An indication that a person has a condition or disease. Some examples of symptoms are headache, fever, fatigue, nausea, vomiting, and pain.

An abnormal mass of tissue that results when cells divide more than they should or do not die when they should. Tumors may be benign (not cancer), or malignant (cancer). Also called neoplasm.

Closely monitoring a patient's condition but withholding treatment until symptoms appear or change. Also called active surveillance, expectant management, and observation.